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BioMarin delays planned FDA filing for hemophilia gene therapy

May 31, 2022

BioMarin’s road back to the FDA keeps getting longer, with the agency’s request adding new uncertainty to a resubmission that’s been in the works for nearly two years.

The California biotech disclosed the FDA’s feedback alongside updated five- and six-year results from an early study of Roctavian, which, if approved, would be the first gene therapy for hemophilia A, the most common form of the inherited bleeding disorder.

Trial participants remained off standard hemophilia treatments and, on average, were experiencing less than one bleeding episode per year — a reduction of more than 90% from prior to treatment.

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